Hey, humans. Today, we talk about the decades-long pursuit to get MDMA approved as a medicine for PTSD, in advance of Lykos Therapeutics’ Sunday PDUFA date. Also, we see a little girl’s ultra-rare disease improve after she was given a genetic medicine designed just for her, and more.
The need-to-know this morning
- Merck acquired an experimental bispecific antibody from China-based Curon Biopharmaceutical for $700 million upfront, plus an additional $600 million in potential milestone payments. The drug, called CN201, is being studied in blood cancers, but Merck said it sees the potential to target B-cell-associated autoimmune diseases.
- Avidity Biosciences said its RNA-based drug produced 25% of normal dystrophin in patients with Duchenne muscular dystrophy caused by a mutation in exon 44.
How a bespoke gene therapy worked
Susannah Rosen, a little girl with a rare and degenerative neurologic disease, received a bespoke treatment about two years ago. Doctors injected her with stretches of genetic code targeting a gene that caused her symptoms into the 8-year-old’s spine, where it could then make its way to her brain. A couple months later, she was able to stand on her own for the first time in four years.
The treatment, based on antisense oligonucleotides, was developed by the San Diego nonprofit n-Lorem. A new Nature study shows that the ASO treatment also reduced her seizures and improved her mobility. That said, she’s still facing vision loss and cognitive difficulties.
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